Samira Kiani, M.D. (PI), leads an eye-opening discussion on the groundbreaking medical advances that could be on the horizon with gene editing. Dr. Kiani is the principal investigator at Kiani lab located on the campus of Arizona State University. She holds a doctorate degree (M.D.) from Tehran University of Medical Sciences. Dr. Kiani gained extensive experience in CRISPR/Cas9 technology while working toward her postdoctorate at the Massachusetts Institute of Technology Center for Integrative Synthetic Biology. Kiani studied the development of synthetic gene circuits used to reprogram various functions and general behavior of mammalian cells based on the advanced CRISPR/Cas9 technology.

As Dr. Kiani explains, gene editing, specifically changing the sequence of DNA is not a new concept; however, the tools used to study gene-editing processes were expensive and perhaps cost prohibitive for some labs. But the CRISPR/Cas9 technology ushered in a new era, as gene editing could now be done rapidly, and at a much lower cost. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. With gene editing, Kiani states that the technology could allow researchers to repair or disable genes. By using CRISPR, Kiani and other researchers are able to work with double-strand DNA breaks.

Dr. Kiani provides an overview of the Cas9 protein, which is a DNA cleaving enzyme that can be manipulated and targeted to any specific DNA sequence via a small guide RNA (gRNA), and it can be adapted for gene editing and gene regulation alike. With the advancing technology, researchers are able to more closely approximate nature, in terms of how genes perform, and enact double-strand breaks in literally any specific location. The potential for CRISPR technology is immense, as the technology would make it possible to potentially provide the correct template to a cell and repair mutations that are causing a particular genetic disease.

The gene editing expert explains that researchers are interested in different forms of CRISPR as each bacteria has its own unique set of CRISPR; thus there is great enthusiasm to learn more about CRISPR that targets RNA, that is simpler, or smaller even. The possibilities are limitless as scientists expand their research, looking for new genes that could fight cancer, disabling genes to understand their specific function, and in vivo experimentation in animals to study how to defeat diseases.

Dr. Kiani discusses in detail what is known in the research world as ‘guide RNA,’ which essentially functions as a select protein’s legs, to take them to the precise location where researchers desire to begin their work. As she explains, the CRISPR technology holds immense promise for human applications but the coming years will see more refinement to address current safety concerns for its uses within humans. As clinical trials are scheduled for everything from blindness to liver disease, gene editing technology experts are extremely hopeful that the answer to complex disease problems could be an edit away.